Regulatory and clinical development to support the approval of advanced therapies medicinal products in Japan.

INTRODUCTION: A new category of products, i.e. regenerative medicine products (RPs), has been defined for advanced therapies medicinal products in Japan, as well as a legislative and regulatory framework to promote their clinical development. AREAS COVERED: This review analyses the most relevant features of the regulatory strategies and clinical development that led RPs to their approval in Japan. EXPERT OPINION: As of 31st September 2021, a total of 14 RPs were approved for 16 indications. From a regulatory standpoint, the available designations allow attractive benefit packages that promote the development of innovative products in Japan and is one of the key points to consider when the global regulatory strategy for the product is being developed. RPs regulations in Japan allow adaptive licensing and constitute shortcut through the clinical development to the approval. RPs have been mainly approved so far based on small studies with inconclusive and limited evidence of efficacy and safety, prioritizing the unmet medical needs of the target diseases, and therefore, the early access for patients. This review also compares the regulatory and clinical development for the current approved RPs in Japan with the development trends in the European Union and United States of America.

Proposal for a regulation on health technology assessment in Europe - opinions of policy makers, payers and academics from the field of HTA.

INTRODUCTION: In January 2018 the European Commission published a Proposal for a Regulation on Health Technology Assessment (HTA): 'Proposal for a Regulation on health technology assessment and amending Directive 2011/24/EU'. A number of stakeholders, including some Member States, welcomed this initiative as it was considered to improve collaboration, reduce duplication and improve efficiency. There were however a number of concerns including its legal basis, the establishment of a single managing authority, the preservation of national jurisdiction over HTA decision-making and the voluntary/mandatory uptake of joint assessments by Member States. Areas covered: This paper presents the consolidated views and considerations on the original Proposal as set by the European Commission of a number of policy makers, payers, experts from pricing and reimbursement authorities and academics from across Europe. Expert commentary: The Proposal has since been extensively discussed at Council and while good progress has been achieved, there are still divergent positions. The European Parliament gave a number of recommendations for amendments. If the Proposal is approved, it is important that a balanced, improved outcome is achieved for all stakeholders. If not approved, the extensive contribution and progress attained should be sustained and preserved, and the best alternative solutions found.